The mission of ALD Connect
is to improve health outcomes for individuals with X-linked adrenoleukodystrophy (ALD) by raising disease awareness, empowering patients, and accelerating the translation of scientific advances into better clinical care. The group was started in the Spring 2013 and just held their first formal annual meeting in Baltimore this week, attended by several hundred people.
ALD Connect is a PPRN (patient powered research network), a hybrid between a patient advocacy group and a traditional medical research consortium. They received funding from the feds (PCORI), from private donors and from industry.
X-ALD is a devastating inherited neuro-metabolic diseases that affects young boys the hardest; but also has a late-onset form that affects women carriers and adult men.
This is the case for co-founder, Ben Lenail, “I am 48 and started developing symptoms around 2007, was diagnosed 2011, and now walk with a cane, among other issues.”
ALD Connect is focused on infrastructure work, such as building a nationwide patient registry, electronic case report forms for natural history studies, and national biomarker database.
“Obviously, we hope this will help us derive insights into potential treatments, and kicking off clinical trials,” says Lenail. “We also enable patients to help each other. I was struck by how many adult patients were well aware, in their hearts of hearts, that they were declining, losing strength, balance and function. They knew they should exercise. But they did not know where to start. This is the spirit of the video we’ve created. We explain what’s wrong with us (our weakness and frailty) and then offer a series of low-impact exercises that have made a huge difference in my own life.”
You can view the video below and learn more about ALD at their website here.